LUM-201 for Growth Hormone Deficiency

(OraGrowtH211 Trial)

Enrolling by invitation at 6 trial locations

Overseen By Lumos Pharma

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Lumos Pharma

Must not be taking: CYP3A4 substrates

Disqualifiers: Genetic conditions, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 7

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial evaluates the long-term safety of LUM-201, a new potential drug for children with Idiopathic Pediatric Growth Hormone Deficiency, a condition where the body doesn't produce enough growth hormone without a clear cause. The trial aims to assess the treatment's effectiveness and the body's response. It is an international study for participants who have already been in a previous LUM-201 growth hormone study for at least 12 months and remain eligible. As a Phase 2 trial, it measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to important research.

Will I have to stop taking my current medications?

You may need to stop taking certain medications that affect the cytochrome system CYP3A4, which is involved in processing LUM-201. If you're on these medications for more than two weeks, you might not be able to participate, but shorter-term use will be evaluated individually.

Is there any evidence suggesting that LUM-201 is likely to be safe for humans?

Studies have shown that LUM-201 is generally well-tolerated. Previous trials revealed no major safety concerns. Participants did not report serious side effects from the treatment. Lab tests, heart checks (ECGs), and other exams showed no significant problems. This suggests that LUM-201 might be safe for people with growth hormone deficiency. However, as this is a Phase 2 trial, closely monitoring safety remains important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about LUM-201 for growth hormone deficiency because it offers a unique approach compared to traditional treatments like daily growth hormone injections. LUM-201 is an oral medication, which is a significant advantage in terms of ease of administration and patient comfort. Additionally, it works by stimulating the body's own natural production of growth hormone, rather than supplying it externally. This novel mechanism could potentially lead to more consistent and physiologically balanced growth hormone levels in patients.

What evidence suggests that LUM-201 might be an effective treatment for growth hormone deficiency?

Studies have shown that LUM-201, the investigational treatment tested in this trial, can significantly increase growth rates in children with growth hormone deficiency. Initial findings suggest that after six months of treatment, children experienced a noticeable increase in growth, which continued with only a slight decrease over 24 months. Research indicates that LUM-201 helps the body produce more of its own growth hormone more effectively than standard treatments. It restores the natural pattern of growth hormone release to levels similar to those in children without this deficiency. Overall, evidence supports the potential of LUM-201 as an effective treatment for children with growth hormone deficiency. ^{3 5 6 7 8}

Are You a Good Fit for This Trial?

This trial is for children with Idiopathic Pediatric Growth Hormone Deficiency who have already been part of a previous LUM-201 study. They must have completed at least 12 months in that study and be cleared to continue treatment. Children with conditions that add risk or those on certain long-term medications affecting LUM-201 metabolism cannot participate.

Inclusion Criteria

You were part of a previous study for pediatric LUM-201 GHD treatment and completed at least the 12-month visit. If you meet all other requirements, you may be able to continue the treatment.

Parent/caregiver must sign the informed consent, and the subject must sign the assent, as applicable.

Exclusion Criteria

I am on long-term medication that affects how my body processes certain drugs.

Medical or genetic condition that, in the opinion of the PI and/or MMs, adds unwarranted risk to the use of LUM-201

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Extension

Participants continue LUM-201 treatment to monitor long-term safety and efficacy

36 months

7 in-person visits with follow-up phone calls between visits

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• LUM-201

Trial Overview The trial is testing the long-term safety of a drug called LUM-201 in children with growth hormone deficiency. It will also look at how the body responds to the drug over time and its effectiveness in promoting growth.

How Is the Trial Designed?

Group I: LUM-201 (1.6 mg/kg/day) Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Lumos Pharma

Lead Sponsor

Trials

5

Recruited

180+

Published Research Related to This Trial

A systematic review of 94 studies indicates that both short-term and long-term growth hormone (GH) replacement therapy in adults with GH deficiency is generally safe, showing no increased risk of diabetes mellitus or cancer recurrence.

Despite the positive findings, the review highlights limitations such as the small number of subjects and insufficient long-term data, which may affect the reliability of the conclusions drawn.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Reviewing the safety of GH replacement therapy in adults. Stochholm, K., Johannsson, G.[2015]

A study involving 135 adults with adult growth hormone deficiency (AGHD) found that both depot growth hormone (GH) given every 14 days and daily GH injections significantly reduced trunk and visceral fat, with the depot form showing a 3.2% decrease in trunk fat compared to no treatment.

Both treatment methods increased lean body mass and had similar side effect profiles, indicating that the depot GH is as safe and effective as daily injections for managing AGHD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of a long-acting growth hormone (GH) preparation in patients with adult GH deficiency. Hoffman, AR., Biller, BM., Cook, D., et al.[2016]

Citations

1. investors.lumos-pharma.com investors.lumos-pharma.com/news-releases/news-release-details/updated-phase-2-oragrowth-data-presented-espe-2024-demonstrate

Updated Phase 2 OraGrowtH Data Presented at ESPE ... Conclusions: LUM-201 shows a significant increase in AHV at 6 months, and the effect continues with a minimal decrease in AHV through 24 months.

2. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9227666/

A GH Secretagogue Receptor Agonist (LUM-201) Elicits ... The current study indicates that LUM-201 can elicit greater GH responses than standard GH secretagogues in some subjects, possibly via its agonist activity on ...

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04614337

Phase 2 Study of LUM-201 in Children With Growth ... This is a multi-national trial. The goals of the trial are to study LUM-201 as a possible treatment for Pediatric Growth Hormone Deficiency (PGHD)

4. academic.oup.com academic.oup.com/jes/article/8/Supplement_1/bvae163.1288/7814116

5162 Oral LUM-201 Restores Pulsatile Growth Hormone ... Conclusions: At 6m LUM-201 was able to restore endogenous GH pulsatile secretion to a similar level to that seen in normal children, while ...

5. globalgenes.org globalgenes.org/raredaily/lumos-reports-positive-topline-data-from-phase-2-studies-of-oral-growth-hormone/

Lumos Reports Positive Topline Data from Phase 2 Studies ... The topline results from the OraGrowtH212 Trial reveal that LUM-201 achieved an expected annualized height velocity with only 20 percent of the ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT05796440

A Long-Term Safety Trial of LUM-201 in Children With ... This is a multi-national trial. The trial aims to study the long-term safety of LUM-201 in subjects with Idiopathic Pediatric Growth Hormone Deficiency (iPGHD).

7. lumos-pharma.com lumos-pharma.com/our-pipeline/lum-201/

LUM-201 LUM-201, also known as ibutamoren, is an investigational, once-daily, orally administered small molecule that promotes the secretion of Growth Hormone (GH) ...

8. endocrine-abstracts.org endocrine-abstracts.org/ea/0110/ea0110p648

Phase 3 pivotal trial design of daily oral LUM-201's efficacy ... LUM-201, an oral growth hormone secretagogue (GHS), acts on the GHS receptor ... Safety assessments will include laboratory tests, ECGs, and pharmacokinetic and ...